المؤلفون: Chorão, Pedro, Henriques, Marta, Villalba, Marta, Montoro, Juan, Balaguer-Roselló, Aitana, González, Eva María, Gómez, María Dolores, Gómez, Inés, Solves, Pilar, Santiago, Marta, Asensi, Pedro, Lamas, Brais, Bataller, Ana, Granados, Pablo, Eiris, Juan, Martínez, David, Louro, Alberto, Rebollar, Paula, Perla, Aurora, Salavert, Miguel, de la Rubia, Javier, Sanz, Miguel Ángel, Sanz, Jaime

المصدر: Transplantation and Cellular Therapy; May 2024, Vol. 30 Issue: 5 p538.e1-538.e10, 48430p

مستخلص: • First CMV reactivations in HSCT with PTCy without letermovir prophylaxis occurred in 46% of patients.• The reactivation rate was 73% for CMV seropositive recipients and 49% for CMV-seropositive donors.• Risk factors for reactivation were CMV seropositivity, haploidentical HSCT, older patient, and grade II-IV acute GVHD.• Second and third CMV reactivations occurred in 13% and 4.4% of patients, independent of donor type.

المؤلفون: Sanz, Miguel Ángel, Montoro, Juan, Balaguer-Roselló, Aitana, Chorão, Pedro, Villalba, Marta, Gómez, Inés, Solves, Pilar, Santiago, Marta, Asensi, Pedro, Lamas, Brais, Bataller, Ana, Granados, Pablo, Eiris, Juan, Martinez, David, Lloret, Pilar, Louro, Alberto, Rebollar, Paula, Perla, Aurora, de la Rubia, Javier, Sanz, Jaime

المصدر: Bone Marrow Transplantation; 20240101, Issue: Preprints p1-11, 11p

مستخلص: This 45-year study (1978–2022) at a single institution evaluated HSCT outcomes and complications, emphasizing recent advances, with to provide insights into HSCT’s evolving field and ongoing efforts to enhance patient outcomes. Involving 1707 patients, the study revealed an initial phase (1978–1987) with a limited activity that yielded modest outcomes, a nearly three-decade span (1988–2016) with a substantial increase in transplant activity, emphasizing umbilical cord blood transplantation (UCBT) for patients lacking a suitable matched sibling donor. In addition to a gradual increase in recipient age, significant improvement in outcomes emerged in the recent period (2017–2022), marked by UCBT replacement with haploidentical transplants, introduction of PTCY-based GVHD prophylaxis for all type of transplants, and increased use of conditioning regimens with thiotepa, busulfan, and fludarabine. In this period, reductions in GVHD, non-relapse mortality, and relapse rates significantly contributed to improved overall survival, event-free survival, and GVHD-free/relapse-free survival. The study identified specific factors, including GVHD prophylaxis and donor selection changes, associated with these positive trends. This four-decade study provides a unique perspective on allogeneic HSCT, showcasing the dynamic evolution of transplantation practices and their impact on outcomes, offering valuable insights for personalized treatment approaches and emphasizing continual innovation in this critical therapeutic modality.

المؤلفون: Izquierdo, Cristina Pascual, Mingot-Castellano, María Eva, Fuentes, Ana E. Kerguelen, García-Arroba Peinado, José, Cid, Joan, Jimenez, Maria Moraima, Valcarcel, David, Gómez-Seguí, Inés, de la Rubia, Javier, Martin, Paz, Goterris, Rosa, Hernández, Luis, Tallón, Inmaculada, Varea, Sara, Fernández, Marta, García-Muñoz, Nadia, Vara, Míriam, Zarzoso, Miguel Fernández, García-Candel, Faustino, Paciello, María Liz, García-García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Estévez, Julia Vidán, Jiménez, Gemma Moreno, López Lorenzo, José Luis, Arias, Elena González, Freiría, Carmen, Solé, María, Ávila Idrovo, Laura Francisca, Hernández Castellet, José Carlos, Cruz, Naylen, Lavilla, Esperanza, Pérez-Montaña, Albert, Atucha, Jon Ander, Moreno Beltrán, María Esperanza, Moreno Macías, Juán Ramón, Salinas, Ramón, del Rio-Garma, Julio

المصدر: Blood Advances; December 2022, Vol. 6 Issue: 24 p6219-6227, 9p

مستخلص: Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by anti-ADAMTS13 antibodies. Caplacizumab is approved for adults with an acute episode of iTTP in conjunction with plasma exchange (PEX) and immunosuppression. The objective of this study was to analyze and compare the safety and efficacy of caplacizumab vs the standard of care and assess the effect of the concomitant use of rituximab. A retrospective study from the Spanish TTP Registry of patients treated with caplacizumab vs those who did not receive it was conducted. A total of 155 patients with iTTP (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5%; P < .05) and less refractoriness (4.5% vs 14.1%; P < .05) than those who were not treated. Time to clinical response was shorter when caplacizumab was used as initial treatment vs caplacizumab used after refractoriness or exacerbation. The multivariate analysis showed that its use in the first 3 days after PEX was associated with a lower number of PEX (odds ratio, 7.5; CI, 2.3-12.7; P < .05) and days of hospitalization (odds ratio, 11.2; CI, 5.6-16.9; P < .001) compared with standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared with the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared with standard of care regimens. When administered within the first 3 days after PEX, it also provided a faster clinical response, reducing hospitalization time and the need for PEX.

المؤلفون: Izquierdo, Cristina Pascual, Mingot-Castellano, María Eva, Fuentes, Ana E. Kerguelen, García-Arroba Peinado, José, Cid, Joan, Jimenez, Maria Moraima, Valcarcel, David, Gómez-Seguí, Inés, de la Rubia, Javier, Martin, Paz, Goterris, Rosa, Hernández, Luis, Tallón, Inmaculada, Varea, Sara, Fernández, Marta, García-Muñoz, Nadia, Vara, Míriam, Zarzoso, Miguel Fernández, García-Candel, Faustino, Paciello, María Liz, García-García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Estévez, Julia Vidán, Jiménez, Gemma Moreno, López Lorenzo, José Luis, Arias, Elena González, Freiría, Carmen, Solé, María, Ávila Idrovo, Laura Francisca, Hernández Castellet, José Carlos, Cruz, Naylen, Lavilla, Esperanza, Pérez-Montaña, Albert, Atucha, Jon Ander, Moreno Beltrán, María Esperanza, Moreno Macías, Juán Ramón, Salinas, Ramón, del Rio-Garma, Julio

المصدر: Blood Advances; December 2022, Vol. 6 Issue: 24 p6219-6227, 9p

مستخلص: •Caplacizumab reduces exacerbation and refractoriness in iTTP.•As initial therapy, caplacizumab accelerates response and reduces the need for PEX and hospital stay.

المؤلفون: Scully, Marie, de la Rubia, Javier, Pavenski, Katerina, Metjian, Ara, Knöbl, Paul, Peyvandi, Flora, Cataland, Spero, Coppo, Paul, Kremer Hovinga, Johanna A., Minkue Mi Edou, Jessica, De Passos Sousa, Rui, Callewaert, Filip, Gunawardena, Sriya, Lin, Julie

المصدر: Journal of Thrombosis and Haemostasis; December 2022, Vol. 20 Issue: 12 p2810-2822, 13p

مستخلص: Caplacizumab demonstrated efficacy and safety in patients with immune‐mediated thrombotic thrombocytopenic purpura (iTTP) in the phase 3 HERCULES trial. However, data on long‐term outcomes following caplacizumab treatment are limited.

المؤلفون: Maia, Catarina, Puig, Noemi, Pérez Ruiz, Cristina, Cedena Romero, Maria Teresa, Guerrero, Camila, Larrayoz, Marta, Botta, Cirino, Gutierrez, Norma C., Calasanz, María José, Martin-Ramos, Maria Luisa, Hernández, Miguel, Rosinol Dachs, Laura, Garcia, Esther González, De Arriba, Felipe, Oriol, Albert, Gonzalez-Calle, Veronica, Escalante, Fernando, de la Rubia, Javier, Gironella Mesa, Mercedes, Rios, Rafael, Garcia Sanchez, Ricarda Belen, Arguiñano PEREZ, Jose Maria, Alegre, Adrian, Martin, Jesus, Couto Caro, María del Carmen, Casanova, Maria, Herraiz, Mario Arnao, Pérez, Ernesto, Garzón López, Sebastián, Gonzalez Perez, Marta Sonia, Martín-Nuñez, Guillermo, Rossi, Adriana, Coleman, Morton, Encinas, Cristina, Vale, Ana M., Teruel, Ana Isabel, Cortés Rodríguez, María, Martinez-Climent, Jose A., Lahuerta, Juan-José, Bladé Creixenti, Joan, Niesvizky, Ruben, San-Miguel, Jesús, Mateos, Maria-Victoria, Paiva, Bruno

المصدر: Blood; November 2022, Vol. 140 Issue: Supplement 1 p4200-4203, 4p

المؤلفون: Maia, Catarina, Puig, Noemi, Pérez Ruiz, Cristina, Cedena Romero, Maria Teresa, Guerrero, Camila, Larrayoz, Marta, Botta, Cirino, Gutierrez, Norma C., Calasanz, María José, Martin-Ramos, Maria Luisa, Hernández, Miguel, Rosinol Dachs, Laura, Garcia, Esther González, De Arriba, Felipe, Oriol, Albert, Gonzalez-Calle, Veronica, Escalante, Fernando, de la Rubia, Javier, Gironella Mesa, Mercedes, Rios, Rafael, Garcia Sanchez, Ricarda Belen, Arguiñano PEREZ, Jose Maria, Alegre, Adrian, Martin, Jesus, Couto Caro, María del Carmen, Casanova, Maria, Herraiz, Mario Arnao, Pérez, Ernesto, Garzón López, Sebastián, Gonzalez Perez, Marta Sonia, Martín-Nuñez, Guillermo, Rossi, Adriana, Coleman, Morton, Encinas, Cristina, Vale, Ana M., Teruel, Ana Isabel, Cortés Rodríguez, María, Martinez-Climent, Jose A., Lahuerta, Juan-José, Bladé Creixenti, Joan, Niesvizky, Ruben, San-Miguel, Jesús, Mateos, Maria-Victoria, Paiva, Bruno

المصدر: Blood; November 2022, Vol. 140 Issue: 1, Number 1 Supplement 1 p4200-4203, 4p

المؤلفون: Mingot Castellano, María Eva, Pascual Izquierdo, Cristina, González, Ataulfo, Viejo Llorente, Aurora, Valcarcel Ferreiras, David, Sebastián, Elena, García Candel, Faustino, Sarmiento Palao, Héctor, Gómez Seguí, Inés, de la Rubia, Javier, Cid, Joan, Martínez Nieto, Jorge, Hernández Mateo, Luis, Goterris Viciedo, Rosa, Fidalgo, Teresa, Salinas, Ramon, del Rio-Garma, Julio

المصدر: Medicina Clínica (ScienceDirect); June 2022, Vol. 158 Issue: 12 p630.e1-630.e14, 56714p

مستخلص: La púrpura trombocitopénica trombótica (PTT) es una microangiopatía trombótica (MAT) caracterizada por el desarrollo de anemia hemolítica microangiopática, trombocitopenia y disfunción orgánica isquémica asociada a niveles de ADAMTS13 inferiores al 10% en la mayoría de los casos.

المؤلفون: Puig, Noemí, Contreras, María-Teresa, Agulló, Cristina, Martínez-López, Joaquín, Oriol, Albert, Blanchard, María-Jesús, Ríos, Rafael, Martín, Jesús, Iñigo, María-Belén, Sureda, Anna, Hernández, Miguel-Teodoro, de la Rubia, Javier, González-Calle, Verónica, Krsnik, Isabel, Cabañas, Valentín, Palomera, Luis, Moraleda, José-María, Bargay, Joan, Cedena, María-Teresa, Paiva, Bruno, Rosiñol, Laura, Bladé, Joan, San Miguel, Jesús, Lahuerta, Juan-José, Mateos, María-Victoria

المصدر: Blood Advances; June 2022, Vol. 6 Issue: 11 p3234-3239, 6p

مستخلص: Monitoring of the monoclonal protein (M-protein) by electrophoresis and/or immunofixation (IFE) has long been used to assess treatment response in multiple myeloma (MM). However, with the use of highly effective therapies, the M-protein becomes frequently undetectable, and more sensitive methods had to be explored. We applied IFE and mass spectrometry (EXENT&FLC-MS) in serum samples from newly diagnosed MM patients enrolled in the PETHEMA/GEM2012MENOS65 obtained at baseline (n = 223), and after induction (n = 183), autologous stem cell transplantation (n = 173), and consolidation (n = 173). At baseline, the isotypes identified with both methods fully matched in 82.1% of samples; in the rest but 2 cases, EXENT&FLC-MS provided additional information to IFE with regards to the M-protein(s). Overall, the results of EXENT&FLC-MS and IFE were concordant in >80% of cases, being most discordances due to EXENT&FLC-MS+but IFE−cases. After consolidation, IFE was not able to discriminate 2 cohorts with different median progression-free survival (PFS), but EXENT&FLC-MS did so; furthermore, among IFE−patients, EXENT&FLC-MS identified 2 groups with significantly different median PFS (P= .0008). In conclusion, compared with IFE, EXENT&FLC-MS is more sensitive to detect the M-protein of patients with MM, both at baseline and during treatment, and provides a more accurate prediction of patients' outcome. This trial was registered at www.clinicaltrials.govas #NCT01916252.

المؤلفون: Puig, Noemí, Contreras, María-Teresa, Agulló, Cristina, Martínez-López, Joaquín, Oriol, Albert, Blanchard, María-Jesús, Ríos, Rafael, Martín, Jesús, Iñigo, María-Belén, Sureda, Anna, Hernández, Miguel-Teodoro, de la Rubia, Javier, González-Calle, Verónica, Krsnik, Isabel, Cabañas, Valentín, Palomera, Luis, Moraleda, José-María, Bargay, Joan, Cedena, María-Teresa, Paiva, Bruno, Rosiñol, Laura, Bladé, Joan, San Miguel, Jesús, Lahuerta, Juan-José, Mateos, María-Victoria

المصدر: Blood Advances; June 2022, Vol. 6 Issue: 11 p3234-3239, 6p

مستخلص: Monitoring of the monoclonal protein (M-protein) by electrophoresis and/or immunofixation (IFE) has long been used to assess treatment response in multiple myeloma (MM). However, with the use of highly effective therapies, the M-protein becomes frequently undetectable, and more sensitive methods had to be explored. We applied IFE and mass spectrometry (EXENT&FLC-MS) in serum samples from newly diagnosed MM patients enrolled in the PETHEMA/GEM2012MENOS65 obtained at baseline (n = 223), and after induction (n = 183), autologous stem cell transplantation (n = 173), and consolidation (n = 173). At baseline, the isotypes identified with both methods fully matched in 82.1% of samples; in the rest but 2 cases, EXENT&FLC-MS provided additional information to IFE with regards to the M-protein(s). Overall, the results of EXENT&FLC-MS and IFE were concordant in >80% of cases, being most discordances due to EXENT&FLC-MS+ but IFE− cases. After consolidation, IFE was not able to discriminate 2 cohorts with different median progression-free survival (PFS), but EXENT&FLC-MS did so; furthermore, among IFE− patients, EXENT&FLC-MS identified 2 groups with significantly different median PFS (P = .0008). In conclusion, compared with IFE, EXENT&FLC-MS is more sensitive to detect the M-protein of patients with MM, both at baseline and during treatment, and provides a more accurate prediction of patients’ outcome. This trial was registered at www.clinicaltrials.gov as #NCT01916252.